Randomized, double-blind, placebo-controlled trial of the efficacy and safety of rilonacept in the treatment of systemic juvenile idiopathic arthritis

Arthritis and Rheumatology

Volumn 66, Issue 9, 2014, Pages 2570-2579

  Ilowite, Norman T ^a   Prather, Kristi ^b   Lokhnygina, Yuliya ^b   Schanberg, Laura E ^c   Elder, Melissa ^d   Milojevic, Diana ^e   Verbsky, James W ^f   Spalding, Steven J ^g   Kimura, Yukiko ^h   Imundo, Lisa F ⁱ   Punaro, Marilynn G ^j   Sherry, David D ^k   Tarvin, Stacey E ^l   Zemel, Lawrence S ^m   Birmingham, James D ⁿ   Gottlieb, Beth S ^o   Miller, Michael L ^p   O'Neil, Kathleen ^l   Ruth, Natasha M ^q   Wallace, Carol A ^r   Singer, Nora G ^s   Sandborg, Christy I ^t   more..

^a ALBERT EINSTEIN COLLEGE OF MEDICINE   (United States)

^b DUKE CLINICAL RESEARCH INSTITUTE   (United States)

^c DUKE UNIVERSITY MEDICAL CENTER   (United States)

^d UNIVERSITY OF FLORIDA   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f MEDICAL COLLEGE OF WISCONSIN   (United States)

^g LERNER RESEARCH INSTITUTE   (United States)

^h HACKENSACK UNIVERSITY MEDICAL CENTER   (United States)

ⁱ NewYork Presbyterian Hospital   (United States)

^j TEXAS SCOTTISH RITE HOSPITAL FOR CHILDREN   (United States)

^k CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^l INDIANA UNIVERSITY HEALTH   (United States)

^m Farmington   (United States)

ⁿ MICHIGAN STATE UNIVERSITY   (United States)

^o Division of Hematology/Oncology and Stem Cell Transplantation   (United States)

^p CHILDREN'S MEMORIAL HOSPITAL   (United States)

^q MEDICAL UNIVERSITY OF SOUTH CAROLINA   (United States)

^r SEATTLE CHILDREN S RESEARCH INSTITUTE   (United States)

^s CASE WESTERN RESERVE UNIVERSITY   (United States)

^t STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

INTERLEUKIN 1; INTERLEUKIN 1 RECEPTOR; PLACEBO; RILONACEPT;

ARTICLE; CHILD; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; FEMALE; HUMAN; JUVENILE RHEUMATOID ARTHRITIS; LOADING DRUG DOSE; MAINTENANCE DRUG DOSE; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SCHOOL CHILD; UNSPECIFIED SIDE EFFECT;

ADOLESCENT; ANTIRHEUMATIC AGENTS; ARTHRITIS, JUVENILE; CHILD; CHILD, PRESCHOOL; DOUBLE-BLIND METHOD; FEMALE; HUMANS; MALE; RECOMBINANT FUSION PROTEINS; TREATMENT OUTCOME;

EID: 84907401360     PISSN: 23265191     EISSN: 23265205     Source Type: Journal    
DOI: 10.1002/art.38699     Document Type: Article

References (34)

1. Grom AA, Passo M. Macrophage activation syndrome in systemic juvenile rheumatoid arthritis. J Pediatr 1996;129:630-2. (Pubitemid 27498267)
2. Singh-Grewal D, Schneider R, Bayer N, Feldman BM. Predictors of disease course and remission in systemic juvenile idiopathic arthritis: significance of early clinical and laboratory features. Arthritis Rheum 2006;54:1595-601.
3. Lomater C, Gerloni V, Gattinara M, Mazzotti J, Cimaz R, Fantini F. Systemic onset juvenile idiopathic arthritis: a retrospective study of 80 consecutive patients followed for 10 years. J Rheumatol 2000;27:491-6. (Pubitemid 30095486)
4. Woo P. Systemic juvenile idiopathic arthritis: diagnosis, management, and outcome. Nat Clin Pract Rheumatol 2006;2:28-34. (Pubitemid 43153646)
5. Spiegel LR, Schneider R, Lang BA, Birdi N, Silverman ED, Laxer RM, et al. Early predictors of poor functional outcome in systemic-onset juvenile rheumatoid arthritis: a multicenter cohort study. Arthritis Rheum 2000;43:2402-9.
6. Sandborg C, Holmes TH, Lee T, Biederman K, Bloch DA, Emery H, et al. Candidate early predictors for progression to joint damage in systemic juvenile idiopathic arthritis. J Rheumatol 2006;33:2322-9. (Pubitemid 44749796)
7. Pascual V, Allantaz F, Arce E, Punaro M, Banchereau J. Role of interleukin-1 (IL-1) in the pathogenesis of systemic onset juvenile idiopathic arthritis and clinical response to IL-1 blockade. J Exp Med 2005;201:1479-86. (Pubitemid 40664091)
8. Verbsky JW, White AJ. Effective use of the recombinant interleukin 1 receptor antagonist anakinra in therapy resistant systemic onset juvenile rheumatoid arthritis. J Rheumatol 2004;31:2071-5. (Pubitemid 39336738)
9. Ruperto N, Quartier P, Wulffraat N, Woo P, Ravelli A, Mouy R, et al. A phase II, multicenter, open-label study evaluating dosing and preliminary safety and efficacy of canakinumab in systemic juvenile idiopathic arthritis with active systemic features. Arthritis Rheum 2012;64:557-67.
10. Ruperto N, Brunner HI, Quartier P, Constantin T, Wulffraat N, Norneff G, et al. Two randomized trials of canakinumab in systemic juvenile idiopathic arthritis. N Engl J Med 2012;367:2396-406.
11. Nigrovic PA, Mannion M, Prince FH, Zeft A, Rabinovich CE, van Rossum MA, et al. Anakinra as first-line disease-modifying therapy in systemic juvenile idiopathic arthritis: report of forty-six patients from an international multicenter series. Arthritis Rheum 2011;63:545-55.
12. Ilowite N, Porras O, Reiff A, Rudge S, Punaro M, Martin A, et al. Anakinra in the treatment of polyarticular-course juvenile rheumatoid arthritis: safety and preliminary efficacy results of a randomized multicenter study. Clin Rheumatol 2009;28:129-37.
13. Zeft A, Hollister R, LaFleur B, Sampath P, Soep J, McNally B, et al. Anakinra for systemic juvenile arthritis: the Rocky Mountain experience. J Clin Rheumatol 2009;15:161-4.
14. Quartier P, Allantaz F, Cimaz R, Pillet P, Messiaen C, Bardin C, et al. A multicentre, randomised, double-blind, placebo-controlled trial with the interleukin-1 receptor antagonist anakinra in patients with systemic-onset juvenile idiopathic arthritis (ANAJIS trial). Ann Rheum Dis 2011;70:747-54.
15. Lequerre T, Quartier P, Rosellini D, Alaoui F, De Bandt M, Mejjad O, et al. Interleukin-1 receptor antagonist (anakinra) treatment in patients with systemic-onset juvenile idiopathic arthritis or adult onset Still disease: preliminary experience in France. Ann Rheum Dis 2008;67:302-8. (Pubitemid 351281262)
16. Economides AN, Carpenter LR, Rudge JS, Wong V, Koehler-Stec EM, Hartnett C, et al. Cytokine traps: multi-component, high-affinity blockers of cytokine action. Nat Med 2003;9:47-52. (Pubitemid 36098259)
17. Lovell DJ, Giannini EH, Reiff AO, Kimura Y, Li S, Hashkes PJ, et al. Long-term safety and efficacy of rilonacept in patients with systemic juvenile idiopathic arthritis. Arthritis Rheum 2013;65:2486-96.
18. Petty RE, Southwood TR, Manners P, Baum J, Glass DN, Goldenberg J, et al. International League of Associations for Rheumatology classification of juvenile idiopathic arthritis: second revision, Edmonton, 2001. J Rheumatol 2004;31:390-2. (Pubitemid 38160763)
19. Feldman B, Wang E, Willan A, Szalai JP. The randomized placebo-phase design for clinical trials. J Clin Epidemiol 2001;54:550-7. (Pubitemid 32453347)
20. Singh G, Athreya BH, Fries JF, Goldsmith DP. Measurement of health status in children with juvenile rheumatoid arthritis. Arthritis Rheum 1994;37:1761-9. (Pubitemid 24371878)
21. Giannini EH, Ruperto N, Ravelli A, Lovell DJ, Felson DT, Martini A. Preliminary definition of improvement in juvenile arthritis. Arthritis Rheum 1997;40:1202-9. (Pubitemid 27430130)
22. Ilowite NT, Sandborg CI, Feldman BM, Grom A, Schanberg LE, Giannini EH, et al. Algorithm development for corticosteroid management in systemic juvenile idiopathic arthritis trial using consensus methodology. Pediatr Rheumatol Online J 2012;10:31.
23. Henter JI, Horne A, Arico M, Egeler RM, Filipovitch AH, Imashuku S, et al. HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer 2007;48:124-31. (Pubitemid 44958267)
24. Wallace CA, Ruperto N, Giannini E, Childhood Arthritis and Rheumatology Research Alliance, Pediatric Rheumatology International Trials Organization, Pediatric Rheumatology Collaborative Study group. Preliminary criteria for clinical remission for select categories of juvenile idiopathic arthritis. J Rheumatol 2004;31:2290-4.
25. O'Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics 1979;35:549-56. (Pubitemid 10242750)
26. De Benedetti F, Brunner HI, Ruperto N, Kenwright A, Wright S, Calvo I, et al. Randomized trial of tocilizumab in systemic juvenile idiopathic arthritis. N Engl J Med 2012;367:2385-95.
27. Stock CJ, Ogilvie EM, Samuel JM, Fife M, Lewis CM, Woo P. Comprehensive association study of genetic variants in the IL-1 gene family in systemic juvenile idiopathic arthritis. Genes Immun 2008;9:349-57. (Pubitemid 351791491)
28. Allantaz F, Chaussabel D, Stichweh D, Bennett L, Allman W, Mejias A, et al. Blood leukocyte microarrays to diagnose systemic onset juvenile idiopathic arthritis and follow the response to IL-1 blockade. J Exp Med 2007;204:2131-44. (Pubitemid 47360874)
29. Macaubas C, Nguyen KD, Peck A, Buckingham J, Deshpande C, Wong E, et al. Alternative activation in systemic juvenile idiopathic arthritis monocytes. Clin Immunol 2012;142:362-72.
30. Mellins ED, Macaubas C, Grom AA. Pathogenesis of systemic juvenile idiopathic arthritis: some answers, more questions. Nat Rev Rheumatol 2011;7:416-26.
31. Lovell DJ, Giannini EH, Reiff A, Cawkwell GD, Silverman ED, Nocton JJ, et al. Etanercept in children with polyarticular juvenile rheumatoid arthritis. N Engl J Med 2000;342:763-9. (Pubitemid 30151920)
32. Ruperto N, Lovell DJ, Quartier P, Paz E, Rubio-Perez N, Silva CA, et al. Abatacept in children with juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled withdrawal trial. Lancet 2008;372:383-91.
33. Gattorno M, Piccini A, Lasiglie D, Tassie S, Brisca G, Carta S, et al. The pattern of response to anti-interleukin-1 treatment distinguishes two subsets of patients with systemic-onset juvenile idiopathic arthritis. Arthritis Rheum 2008;58:1505-15. (Pubitemid 351705940)
34. Kapur S, Bonk ME. Rilonacept (arcalyst), an interleukin-1 trap for the treatment of cryopyrin-associated periodic syndromes. P T 2009;34:138-41.